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The rising impact of biomarkers in early clinical development

Drug Target Review

A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. Diagnostic biomarkers typically confirm or establish a diagnosis and are often used in selecting patient populations for clinical trials.

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Biogen Advances Investigational SMA Therapy to Registrational Trials After Positive Phase 1 Data

The Pharma Data

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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Amgen Unveils Phase 2 Obesity Trial Results for Monthly Maritide at ADA’s 85th Scientific Sessions

The Pharma Data

In tandem with the Phase 2 results, Amgen also shared detailed data from its Phase 1 Pharmacokinetics Low Dose Initiation (PK-LDI) study. Safety Profile and Tolerability: No Surprises, GI Events Mostly Mild In terms of safety, the Phase 2 study did not identify any new safety signals.

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UCB’s GEMZ Phase 3 Trial Shows Promising Results for Fenfluramine in Treating CDKL5 Deficiency Disorder

The Pharma Data

The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. The most common adverse events were mild to moderate in intensity and did not differ significantly from known safety patterns.

Trials 40
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Gamcemetinib

New Drug Approvals

Gamcemetinib CAS 1887069-10-4 CC-99677 , OS2IR8TV1O Molecular Weight 469.94 nM) and cell based assays ( EC 50 =89 nM).

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Novo Nordisk: Phase 3 Data Shows Mim8 Well-Tolerated After Switch from Emicizumab in Hemophilia A

The Pharma Data

The study marks a significant step forward in the treatment landscape for haemophilia A, demonstrating not only clinical safety and pharmacokinetic stability, but also strong patient preference for the Mim8 pen-injector delivery system. In the trial, participants were directly switched to Mim8 without a washout period.

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Orforglipron’

New Drug Approvals

1] [3] Clinical trials The results of Phase I safety and Phase II ascending-dose clinical trials enrolling people with obesity or type 2 diabetes were published in 2023. [4] “Eli Lilly’s weight loss pill succeeds in first late-stage trial on diabetes patients” CNBC. then was licensed to Lilly in 2018. [1]